$1.5 Million NIH SBIR Grant to Support Development of Rheumatoid Arthritis Vaccine

Rheumatoid Arthritis is a chronic inflammatory disease that mainly targets the synovial membrane, cartilage and bone. It affects about 1% of the global population and is associated with significant morbidity and increased mortality.

Anti-TNF related therapies are the current standard treatment of patients with advanced RA, but over half of the RA patients do not respond to current anti-TNF drugs such as etanercept (Enbrel®) and infliximab (Remicade®).

CEL-SCI Corporation has been awarded a new Phase II Small Business Innovation Research (SBIR) grant by the National Institute of Arthritis and Musculoskeletal and Skin Diseases entitled “Preclinical studies of PG70 LEAPS peptide vaccine for rheumatoid arthritis” in the amount of approximately $1.5 million, that will provide funding to allow CEL-SCI to advance its first LEAPS product candidate, CEL-4000, towards an Investigational New Drug (IND) application, by funding GMP manufacturing, IND enabling studies, and additional mechanism of action studies. Previous pre-clinical studies show CEL-SCI’s LEAPS technology prevented the development, lessened the severity of arthritis and described in part the mechanism of action and target cells

The work will be conducted at CEL-SCI’s research laboratory and Rush University Medical Centerin Chicago, Illinois at the laboratories of Tibor Glant, MD, Ph.D., The Jorge O. Galante Professor of Orthopedic Surgery and Katalin Mikecz, MD, Ph.D. Professor of Orthopedic Surgery & Biochemistry.

The grant was awarded based on published data by Dr. Glant’s team in collaboration with CEL-SCI showing that the administration of a proprietary peptide using their LEAPS technology prevented the development, and lessened the severity, including inflammation, of experimental proteoglycan induced arthritis (PGIA or GIA) when it was administered after the disease was induced in the animals. This data was recently published in Vaccine in an article titled “An epitope-specific DerG-PG70 LEAPS vaccine modulates T cell responses and suppresses arthritis progression in two related murine models of rheumatoid arthritis” by Mikecz et al.

“These findings, in conjunction with the results from previously conducted studies with LEAPS vaccines in the PGIA and GIA and other autoimmune models suggest that LEAPS vaccines may be used as a therapeutic treatment in a variety of different types of autoimmune conditions. LEAPS vaccines may be advantageous to other therapies because the LEAPS vaccines act early on the immune system and inhibit the production of disease-promoting inflammatory cytokines, unlike anti-Tumor necrosis factor alpha (TNFa) therapy which generally acts late and neutralizes only one individual inflammatory cytokine out of many involved in the disease process,” said CEL-SCI’s Senior Vice President of Research, Daniel Zimmerman, Ph.D.

Dr. Zimmerman continued, “The successful conclusion of this round of studies in this autoimmune disease takes LEAPS closer to human studies and could open its development to various other autoimmune diseases, such as multiple sclerosis, uveitis, colitis (inflammatory bowel disease) and certain types of diabetes.”

CEL-SCI’s work is focused on finding the best way to activate the immune system to fight cancer and infectious diseases. Its lead investigational immunotherapy, Multikine* (Leukocyte Interleukin, Injection), is currently being studied in a pivotal Phase 3 clinical trial as a potential neoadjuvant treatment for patients with squamous cell carcinoma of the head and neck. The study was designed with the objective that, if the study endpoint, which is an improvement in overall survival of the subjects treated with the Multikine treatment regimen plus the current standard of care (SOC) as compared to subjects treated with the current SOC only, is satisfied, the study results will be used to support applications that the Company plans to submit to regulatory agencies in order to seek commercial marketing approvals for Multikine in major markets around the world.

The Company’s LEAPS technology is being developed as a therapeutic vaccine for rheumatoid arthritis and is supported by grants from the National Institutes of Health.